Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized

Abstract

Summary. The frequency of joint bleeds and orthopaedic joint scores were evaluated in 121 patients with severe haemophilia who had started prophylactic treatment with clotting factor concentrates at least once weekly before the age of 10. 75 of the patients started before the age of 3, 31 at the age of 3-5 and 15 at the age of 6-9. Each subgroup was evaluated separately. In addition, a regimen of one infusion weekly was compared with that of two (haemophilia B) or three (haemophilia A) infusions weekly in each patient.A significant decrease in the overall number of joint bleeds per year was found after shortening the infusion interval (P < 0·005), but the individual bleeding pattern varied. In survival analysis of the first pathologic joint score event, those who started prophylaxis before the age of 3 had a better outcome overall than those starting at later ages (P ¼ 0·001). However, in subgroup analysis, no significant difference was seen in the annual number of joint bleeds and the development of arthropathy between those starting with, or shifting to, the more intensive regimen before the age of 3 and those that were put on this regimen at the age of 3-5. Age at start of prophylaxis was found to be an independent predictor for the development of arthropathy (P ¼ 0·0002), whereas dose and infusion interval at start were not.Our data emphasize the importance of starting replacement therapy during the first years of life. However, it seems that when beginning the regimen it can be individualized and adjusted according to the bleeding pattern. In this way, the need for a venous access system may be assessed on an individual basis.

Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia

“…The potential for prophylaxis to alter the natural history of severe haemophilia has been demonstrated in retrospective cohort studies [23][24][25][26] and a prospective randomized study [27]. However, debate continues regarding the exact timing and optimal prophylaxis regimen for patients with severe haemophilia A.…”

Hemophilia severity score system: validation from an Italian Regional Hemophilia Reference Center

“…hemophilia [7,8]. It is therefore possible that a large proportion of their patients on demand therapy were mild bleeders.…”

Recombinant full‐length factor VIII (FVIII) and extended half‐life FVIII products in prophylaxis – new insight provided by pharmacokinetic modelling

“…Prophylactic, regular and prolonged administration of clotting factor concentrates represents the mainstay of modern haemophilia management since it has been proven to be effective in preventing bleeding and thus development of haemophilic arthropathy [1][2][3]. This premise is based on the observation that patients with mild/moderate haemophilia (i.e.…”